A Study of Emapalumab for Pediatric Aplastic Anemia

Recruiting now · Phase 2

Conditions studied: Aplastic Anemia, Cytopenia, Hypocellular Marrow

In brief

The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options. Funding Source- FDA OOPD

Key facts

Study ID

NCT06430788

Run by

Memorial Sloan Kettering Cancer Center

People needed

35

Starts

2024-05-21

Expected to finish

2029-05-21

Last updated by the study team

2025-12-17

Who can join

Age: any, up to 25. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• Patients undergoing workup for suspected newly diagnosed sAA:
• Patients with severe cytopenias and a hypocellular marrow concerning for sAA
• Patients that meet the definition for suspected sAA (Camitta Criteria) as follows:
• Marrow Cellularity: <25%, or 25-50% with <30% residual hematopoietic cells Peripheral cytopenias (at least 2 of 3) Absolute neutrophil count (ANC): <500 x 10\^9/L Platelets: <20 x 10\^9/L Absolute Reticulocyte Count: <60 x 10\^9/L
• Patients that do not have evidence of leukemia or MDS
• Patients < 25 years of age at time of diagnosis
• Able to tolerate emapalumab and IST (with standard institutional organ function criteria)

You may not qualify if…

• Uncontrolled infection at presentation.
• Patients who have undergone previous treatment for sAA.
• Patients with known inherited bone marrow failure
• Patient who has completed a full workup for sAA including having results back from telomere testing, DEB and genetics (when applicable), as well as having an appropriate willing and available donor and would otherwise be admitted for HSCT within 2 weeks of enrolling on the trial
• Patients with leukemia or MDS
• Patient or parent or guardian unable to give informed consent or unable to comply with the treatment protocol including research tests.

Where it is running

• Memorial Sloan Kettering Cancer Center (All Protocol Activities) — New York, New York, United States (enrolling)
• Cincinnati Children's Hospital Medical Center (Data collection only) — Cincinnati, Ohio, United States (enrolling)
• Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis) — Philadelphia, Pennsylvania, United States (enrolling)
• Virginia Commonwealth University (Data Collection Only ) — Richmond, Virginia, United States (enrolling)
• Children's Hospital of Wisconsin (Data Collection Only) — Milwaukee, Wisconsin, United States (enrolling)
• Medical College of Wisconsin (Data Collection AND Data Analysis) — Milwaukee, Wisconsin, United States

Full record on ClinicalTrials.gov

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